Cytokinetics announced that it has received feedback from the FDA that the six minute walk test, or 6MWT, is an acceptable primary efficacy endpoint for a potential registration program for reldesemtiv in patients with spinal muscular atroph, or SMA, who have maintained ambulatory function. The FDA also recommended adding a global function scale as a secondary endpoint, such as the Hammersmith Functional Motor Scale, or HFMSE. In collaboration with Astellas, Cytokinetics is developing reldesemtiv as a potential treatment for people with SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue. The companies are discussing potential next steps regarding the ongoing development program for reldesemtiv in this patient population. Results from a Phase 2 clinical study of reldesemtiv in patients with SMA showed a statistically significant exposure-response relationship in changes from baseline in six minute walk distance, or 6MWD, a sub-maximal exercise test of aerobic capacity and endurance. The study also showed statistically significant increases for maximal expiratory pressure, or MEP, a measure of strength of respiratory muscles. Other assessments, including the HFMSE, did not demonstrate differences between reldesemtiv versus placebo. Adverse events in the Phase 2 study in patients with SMA were similar between treatment groups receiving reldesemtiv and placebo. Cytokinetics is preparing to conduct an additional Phase 1 study of reldesemtiv in healthy volunteers designed to assess whether higher doses than were evaluated in the Phase 2 study of patients with SMA may result in higher plasma concentrations. This study is expected to begin in Q1.
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